SIRION Biotech and Denali Therapeutics Join Forces to Develop Gene
Therapies for Diseases of the Central Nervous System

January 09, 2019 08:30 AM Eastern Standard Time

MUNICH--(BUSINESS WIRE)--SIRION Biotech GmbH, a world leader in viral vector-based gene delivery
technologies for gene & cell therapy, announced today that it signed a
license and collaboration agreement with Denali Therapeutics (NASDAQ:
DNLI), a biopharmaceutical company developing a broad portfolio
of product candidates for neurodegenerative diseases, to develop
adeno-associated virus (AAV) vectors to enable therapeutics to cross the
blood-brain barrier for clinical applications in neurodegenerative
diseases including Parkinson’s, Alzheimer’s disease, ALS and certain
other diseases of the CNS. Once again SIRION’s strategic alliance
partner Prof. Dr. Dirk Grimm from the University Hospital Heidelberg
(UKHD) is a key contributor to this highly innovative development. Main
terms of the agreement include development expenses, upfront and
contingent milestone payments to SIRION Biotech, and additional
low-to-mid single digit royalties on net sales resulting from certain
future products or therapies.

AAV vectors are considered the most promising gene delivery system for
therapeutic applications. Denali Therapeutics, together with SIRION
Biotech and Dirk Grimm, a world-renowned and pioneering scientist in the
field of AAV biology and application, will combine their proprietary
technology platforms to create a next generation of AAV vectors. The
goal is to develop new and modified AAV capsids exhibiting a safe
product profile with improved specificity and high efficiency for
therapeutic drug delivery to the brain at therapeutic levels to
effectively treat patients. “This ground-breaking collaboration will
help Denali Therapeutics to increase the availability of protein
therapeutics in the brain, and to quickly enter clinical trials with
efficient, safe and scalable therapeutic candidates”, said Dr. Christian
Thirion, founder and CEO of the company. “We believe that through our
partnership with Denali, CNS-directed AAV-based gene therapies can reach
the market in the fastest possible way, providing novel treatment
options to many millions of patients worldwide suffering from
devastating neurodegenerative diseases such as Parkinson’s, Alzheimer’s,
and ALS,” added Dr. Sabine Ott, VP BD & Licensing. Prof. Dirk Grimm
notes that "By contributing and harnessing our unique and proprietary
expertise in the engineering and high-throughput in vivo screening of
AAV capsid libraries, we will significantly accelerate this joint
endeavour and increase our chances to realize its pivotal aims."

Additionally, Alexander Schuth, COO of Denali Therapeutics stated, “We
are excited to partner with SIRION Biotech and combine our expertise
around the blood-brain barrier and neurodegenerative diseases with
SIRION’s leading expertise on viral vectors for gene therapy to enable
new treatments for diseases of the brain. This partnership will add a
new therapeutic modality to our portfolio and is complementary to our
existing biotherapeutic and small molecule programs.”

About SIRION Biotech GmbHSIRION Biotech was founded in 2005
with the goal to spark a new generation of viral vector technologies for
gene and cell therapy as well as vaccine development. SIRION evolves
novel therapeutic viral vectors and uses proprietary technology
platforms based on lenti-, adeno-, and adeno-associated viruses, to
expedite its partners’ advances in drug development. For additional
information, please visit www.sirion-biotech.com.

About Denali Therapeutics Inc.Denali is a biopharmaceutical
company developing a broad portfolio of product candidates for
neurodegenerative diseases. Denali pursues new treatments by rigorously
assessing genetically validated targets, engineering delivery across the
blood-brain barrier and guiding development with biomarker monitoring to
demonstrate target engagement and select patients. Denali is based
in South San Francisco. For additional information, please visit www.denalitherapeutics.com